An international, open-label Phase 3 study, co-led by Susanna McColley, MD, from Ann & Robert H. Lurie Children’s Hospital of Chicago, found that a regimen of three drugs (elexacaftor/tezacaftor/ivacaftor) that targets the genetic cause of cystic fibrosis was safe and effective in 6-11-year-olds with at least one copy of F508del...
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DUBLIN, Ireland — Theravance Biopharma Inc. today announced results from the Phase 4 PIFR-2 study of YUPELRI® (revefenacin) inhalation solution, the only once-daily, nebulized long-acting muscarinic antagonist (LAMA) approved in the U.S. for maintenance treatment of COPD. The PIFR-2 study aimed to demonstrate greater improvement in lung function for YUPELRI...
SAN ANTONIO, TX — A ribbon of brain tissue called cortical gray matter grows thinner in people who go on to develop dementia, and this appears to be an accurate biomarker of the disease five to 10 years before symptoms appear, researchers from The University of Texas Health Science Center...
Wellington, New Zealand – The Malaghan Institute of Medical Research in collaboration with Wellington Zhaotai Therapies Limited today announced results of its phase 1 dose escalation trial of a new third generation anti-CD19 chimeric antigen receptor (CAR) T-cell therapy was presented at the American Society of Hematology (ASH) Annual Meeting...
ATLANTA — The first-ever national estimate among a nationally representative sample of U.S. children revealed that 3 out of every 1,000 children between the age of 6 and 17 in the United States have been diagnosed with Tourette Syndrome (TS), according to a study by the Centers for Disease Control...
HELSINKI, Finland — TILT Biotherapeutics (TILT), a clinical-stage biotechnology company developing cancer immunotherapies, announces it has been selected by the U.S. Department of Defense (DOD), America’s largest government agency, to receive a USD 2M grant for a three-year project on treatment for ovarian cancer using the company’s TILT-123 asset. The...
Johns Hopkins brain scientists have figured out why a faulty protein accumulates in cells everywhere in the bodies of people with Huntington’s disease (HD), but only kills cells in the part of the brain that controls movement, causing negligible damage to tissues elsewhere. The answer, reported this week in Science,...
Gothenburg, Sweden – Proteins that form clumps occur in many difficult-to-treat diseases, such as ALS, Alzheimer’s, and Parkinson’s. The mechanisms behind how the proteins interact with each other are difficult to study, but now researchers at Chalmers University of Technology, Sweden, have discovered a new method for capturing many proteins...
COPENHAGEN, Denmark — Genmab A/S (Nasdaq: GMAB) and Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) seeking to convert the accelerated approval of TIVDAK® (tisotumab vedotin-tftv) to full approval, for the treatment of patients with recurrent or metastatic...
ST. LOUIS, MO – Each year, tuberculosis (TB) kills more people than any other infectious disease, falling out of the top spot only temporarily during the COVID-19 pandemic. Despite TB’s wide reach and some lost progress during the COVID-19 pandemic, researchers believe it is possible to eradicate TB through advances...